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MOD-1301 is a long-acting recombinant protein therapeutic developed by OPKO Biologics for the treatment of severe primary insulin-like growth factor 1 deficiency (SPIGFD). The drug utilizes proprietary Carboxyl Terminal Peptide (CTP) technology, which involves the fusion of CTP sequences derived from the human chorionic gonadotropin (hCG) beta subunit to the IGF-1 protein. This modification is designed to significantly extend the circulatory half-life of the therapeutic, potentially reducing the injection frequency required for hormone replacement therapy compared to standard daily IGF-1 formulations. MOD-1301 acts as an agonist at the IGF-1 receptor (IGF1R), promoting linear growth and addressing metabolic abnormalities in patients with SPIGFD, a condition characterized by extreme short stature and resistance to growth hormone. While the MOD-1301 program was discontinued, a related asset, OPK-8801002, remains in preclinical development for the same indication.
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